That $930M drug Alexion bought to pair with Soliris, Ultomiris? AstraZeneca says it just aced a PhIII test

16 Sep 2022
endpts.com
Back in 2019, Alexion paid $930 million to snap up Achillion and danicopan, its promising companion drug to Soliris. Three years later, Alexion itself became a subsidiary of AstraZeneca — which is taking the honors Friday morning to announce an interim Phase III success.
The drug met its primary endpoint of change in hemoglobin among patients with paroxysmal nocturnal hemoglobinuria who experience clinically significant extravascular hemolysis, when given on top of either Soliris or the follow-on Ultomiris.
Alexion pays $930M to buy out Achillion and its promising companion drug to Soliris
While C5 inhibitors are effective in treating the rare blood disorder, a small percentage may continue to experience anemia, said Jong-Wook Lee, a professor in Korea and an investigator of the Phase III ALPHA trial.
“These data show that danicopan has the potential to resolve clinically significant EVH while allowing patients to remain on standard of care treatment with Ultomiris or Soliris,” he added.
In addition to measuring change in hemoglobin from baseline at 12 weeks, the study was also designed to track transfusion avoidance and change in the fatigue score based on the Functional Assessment of Chronic Illness TherapyChronic Illness Therapy.
According to AstraZeneca, danicopan plus Ultomiris or Soliris beat placebo on all those aspects for this specific patient group.
This is not the only use it has in mind for the drug, which is an oral factor D inhibitor. As Alexion’s former CEO Ludwig Hantson previously explained, factor D inhibition tackles an alternative pathway to C5 and “addresses uncontrolled complement activation further upstream in the complement cascade, and importantly, leaves the rest of the complement system intact.”
Alexion is also testing danicopan as a monotherapy for geographic atrophy in a Phase II clinical trial.
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